A Road Map for Biomedical Frontier

The NIH, seeking to bring together experts from many specialties to speed discoveries, unveils a plan for funding, experiments.

WASHINGTON — Saying that medical breakthroughs are coming less frequently from lone scientists in individual laboratories, the National Institutes of Health on Tuesday unveiled a $2-billion plan that would encourage biologists, computer experts, physicists and other specialists to work together and would provide them with new tools to conduct experiments.

"This is truly not business as usual for medical research," said Dr. Elias A. Zerhouni, director of the NIH. The goal, he said, is to move laboratory discoveries more quickly to patients.

With a budget of $27 billion, the NIH funds a huge array of experiments at universities, hospitals and its own institutions.

Zerhouni said the agency would spend $2.1 billion over five years on its new "road map for medical research," a collection of 28 initiatives designed to accelerate scientific discoveries.

The proposals attempt to deal with the recognition that most biological research is no longer carried out simply by a single scientist with a test tube and a microscope.

To study how dozens of genes interact to cause disease, for example, biologists must use computer software and work with data management experts. To create new ways of taking pictures of cells, they work with physicists. To create materials that help cells to grow into artificial organs, they work with engineers and materials specialists.

"The NIH roadmap is being driven by changes in the science. We need changes in our community to adjust to 'big science' from what has always been a cottage industry," said Dr. David Korn, senior vice president for research at the Assn. of American Medical Colleges.

The NIH proposal calls for new funding for research and training programs that include multiple specialties.

It also aims to build shared collections of research tools that are common in pharmaceutical companies but remain largely unavailable to university and hospital researchers.

For example, the NIH wants to build a large library of molecules that might turn out to be useful drugs, along with new centers with automated equipment that can test those molecules for their effect on genes and cells.

Currently, for example, a scientist who determines that a specific gene is linked to Parkinson's disease has few ways to explore whether that discovery can lead to a treatment, said Dr. Francis Collins, who leads the NIH's National Human Genome Research Institute.

In the future, that researcher would be able to tap the NIH molecule library and automated machinery to try to find molecules that alter the workings of that gene, Collins said. Some of them may yield new clues about how the gene works or even point toward a treatment.

Some of the NIH proposals are aimed at improving research on patients. The agency wants to connect traditional community-based doctors with researchers at hospitals and universities who test new treatments on patients.

The NIH hopes that this will allow researchers to set up new studies more rapidly and that more patients will be drawn into the studies. Only 3% to 4% of cancer patients, for example, currently enroll in studies on new treatments, pain management and other elements of the illness, Zerhouni said.

The NIH will also make at least 10 grants annually to scientists pursuing ideas considered creative but risky -- a response to complaints that the agency backs only "safe" proposals. Each grant would offer $2.5 million over five years.

"NIH has always been guilty of not enough blue-sky thinking, of being too rigid on how you have to do lots of experiments on something before you can win support for it," said Dr. Leroy Hood, president of the Institute for Systems Biology, a nonprofit independent research institution in Seattle.

For example, Hood said, the NIH rejected two grant applications he made in the 1980s for the first gene-sequencing machines, which later helped revolutionize biology. The agency is "focused on the present and not on the future ... and so this new element is a terrific addition to what the NIH already does well," he said.

As an example of a high-risk proposal it might support, the NIH cited "preventive biosensors" that would scan the body for molecular signs of disease and eliminate them. While ideas like this "might read like science fiction, they are much closer to reality than one might imagine," the agency said.

Korn said that universities and the NIH would have to make "cultural changes" to encourage scientists to join large research groups.

Scientists often want to work alone or in small groups so they can be the principal author of papers that announce significant discoveries. Universities give tenure, and the NIH gives grants, to scientists who are lead authors of important papers.

Korn said scientists would need assurance that if they joined a large group, they would be rewarded even for being the fourth or fifth author on a paper. "This will affect how universities evaluate scholarship and make tenure decisions," he said.